Spice turmeric corrects the cystic fibrosis defect

Scientists at The Hospital for Sick Children (Sick Kids) and Yale University School of Medicine have found that a compound in the spice turmeric corrects the cystic fibrosis defect in mice. This research is reported in the April 23, 2004 issue of the journal Science.

Cystic fibrosis (CF) is a genetic disease that affects approximately 30,000 Americans (70,000 worldwide) and causes the body to produce an abnormally thick, sticky mucus that accumulates in the lungs and digestive system. People with CF have difficulty absorbing nutrients from food and develop recurrent lung infections. These lung infections and the subsequent pulmonary damage are often fatal. The median age of survival for people with CF is in the early thirties.

The laboratories of Drs. Marie Egan, Michael Caplan (both at Yale University School of Medicine), and Gergely Lukacs (Sick Kids) demonstrated in a mouse model that curcumin treatment can release the mutant CFTR protein from this inappropriate compartment inside the cell and allow it to reach its proper destination, where it is able to function. Furthermore, oral curcumin treatment was able to correct characteristic cystic fibrosis defects in a mouse model of the disease. Curcumin is a compound found in turmeric, and is what gives the spice its bright yellow colour and strong taste.

“We were able to prove at the cellular level what the Yale group observed in the mouse model of the disease,” said Dr. Gergely Lukacs, a senior scientist in the Cell and Lung Biology Research Programs at Sick Kids and associate professor in the Department of Laboratory Medicine and Pathobiology at the University of Toronto. “After having received curcumin treatment, mice with the genetic defect that causes CF survived at a rate almost equal to normal mice. The CFTR protein also functioned normally in the cells lining the nose and rectum, which are areas of the body affected by cystic fibrosis.”

Dr. Michael Caplan, the study’s senior author and professor in the Department of Cellular and Molecular Physiology at Yale University School of Medicine, said: “In the next phase of this research, we will work to determine precisely how curcumin is achieving these effects and to optimize its potential as a possible drug. Plans are underway for a human clinical trial of curcumin, which will be carried out under the auspices of Cystic Fibrosis Foundation Therapeutics, Inc.”

Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation (US), is working with Seer Pharmaceuticals on a Phase I clinical trial of curcumin to assess safety and dosage parameters in humans. The trial will be conducted through CFFT’s Therapeutics Network in four to six sites and will include approximately 25 patients.

This research was supported by the Canadian Cystic Fibrosis Foundation, the Canadian Institutes of Health Research, The Hospital for Sick Children Foundation, Alyward Family/Pitney Bowes Gift Fund, the US Cystic Fibrosis Foundation, the US National Institutes of Health, and a sponsored research grant from Seer Pharmaceuticals to Yale University.

The Hospital for Sick Children, affiliated with the University of Toronto, is Canada’s most research-intensive hospital and the largest centre dedicated to improving children’s health in the country. Its mission is to provide the best in family-centred, compassionate care, to lead in scientific and clinical advancement, and to prepare the next generation of leaders in child health. For more information, please visit www.sickkids.ca.

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